Tonix Pharmaceuticals to Present at B. Riley Securities’ 2022 Virtual Neuro & Ophthalmology Conference

Research, News, and Market Data on Tonix Pharmaceuticals

CHATHAM, N.J., April 25, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, announced today that Seth Lederman, President and Chief Executive Officer of Tonix Pharmaceuticals, will present at B. Riley Securities’ 2022 Virtual Neuro & Ophthalmology Conference on Wednesday, April 27, 2022, at 1:00 p.m.ET.

A webcast of the presentation will be available under the IR Events tab of the Tonix website at www.tonixpharma.com.

About Tonix Pharmaceuticals Holding Corp.
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500¹ which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-2900² for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-801³, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-1850⁴, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-3500⁵ (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL⁶, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-1300⁷ is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.

¹TNX-1500 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
²TNX-2900 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
³TNX-801 is an investigational new biologic at the pre-IND stage of development and has not been approved for any indication.
⁴TNX-1840 and TNX-1850 are investigational new biologics at the pre-IND stage of development and have not been approved for any indication. 
⁵TNX-3500 is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
⁶TNX-102 SL is an investigational new drug and has not been approved for any indication.
⁷TNX-1300 is an investigational new biologic and has not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 799-8599

Olipriya Das, Ph.D. (media)
Russo Partners
Olipriya.Das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Ocugen, Inc. Announces Positive DSMB Recommendation For OCU400-101 Clinical Trial

Research, News, and Market Data on Ocugen

PHASE 1/2 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU400 MODIFIER GENE THERAPY CANDIDATE TO TREAT RETINITIS PIGMENTOSA ASSOCIATED WITH NR2E3 AND RHO MUTATIONS

MALVERN, Pa., April 25, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines, announced today that the independent Data and Safety Monitoring Board (DSMB) for its Phase 1/2 clinical trial of OCU400, the Company’s flagship modifier gene therapy candidate for the treatment of Retinitis Pigmentosa (RP), reviewed safety data based on dosing to date and recommended that the study proceed with enrolling additional subjects.

The OCU400-101 clinical study to assess the safety and efficacy of modifier gene therapy candidate OCU400 for RP resulting from mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes recently dosed its first patient. The DSMB recommended that the Company continue enrolling the remaining study subjects in this current cohort at the target dose level.

Ocugen’s modifier gene therapy platform targets nuclear hormone receptors (NHRs) that regulate multiple functions within the retina, giving it the potential to address many different gene mutations – and in turn, multiple retinal diseases – with a single product. Traditional gene therapy, which transfers a functional version of a non-functional gene into target cells, addresses only one individual gene mutation at a time.

“It’s a positive first step that the DSMB review of the current OCU400-101 study results identified no serious adverse events and recommended that the study proceed with enrollment,” said Mark Pennesi, MD, PhD, Professor of Ophthalmology and Chief of the Paul H. Casey Ophthalmic Genetics Division, Oregon Health & Science University, and member of Ocugen’s Retina Scientific Advisory Board. “We’re looking forward to understanding how this modifier gene therapy platform could treat inherited retinal degeneration, potentially bringing an option to people affected with this disease.”

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines that improve health and offer hope for people and global communities. We are making an impact through courageous innovation, taking science in new directions in service of patients. Our breakthrough modifier gene therapy platform has the potential to treat multiple diseases with one drug and we are advancing research in other therapeutic areas to offer new options for people with unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include information about qualitative assessments of available data, potential benefits, expectations for clinical trials, and anticipated timing of clinical trial readouts and regulatory submissions, including with respect to our Phase 1/2 trial included in our Investigational New Drug application to the U.S. Food and Drug Administration (FDA) for OCU400, which is actively enrolling patients following review of preliminary safety data by the independent Data and Safety Monitory Board. This information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; the risk that clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; and the risk that the Orphan Drug Designations from the FDA and broad Orphan Medicinal Product Designation from the European Commission for OCU400 may not result in a faster approval timeline for OCU400 or increase the likelihood of any such approvals These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Ocugen Contact:
Ken Inchausti
Head, Investor Relations & Communications
ken.inchausti@ocugen.com

Please submit investor-related inquiries to: IR@ocugen.com

Lineage Announces A Fifth Cell Therapy Program: Allogeneic Photoreceptor Transplants For The Treatment Of Diseases Which May Lead To Blindness

Research, News, and Market Data on Lineage Cell Therapeutics

Dynamic Culturing Process Developed by Lineage Offers Path to Clinical- and Industrial-Scale Production of Photoreceptors

CARLSBAD, Calif.–(BUSINESS WIRE)–Apr. 25, 2022– 

Lineage Cell Therapeutics, Inc.
 (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced a new cell therapy development program: photoreceptor neural cell (PNC) transplants for the treatment of vision loss due to photoreceptor dysfunction or damage. Similar to the company’s recently announced pipeline expansion into auditory neurons for the treatment of hearing loss, Lineage has filed for intellectual property protection covering the composition and methods for generating PNCs. Based on recent in vivo data generated using the company’s PNCs, these cells may be capable of forming reconstructed retina with high survivability and neural connectivity to surrounding functional layers. Notably, Lineage has demonstrated feasibility which could support a large-scale method for producing both types of photoreceptors, known as rods and cones.

“It is natural that, on the heels of the announcement of our alliance with Roche and 
Genentech for our RPE cell therapy, a deal worth up to 
$670 million dollars plus double-digit royalties if certain development, approval, and sales milestones are achieved and other conditions are met, that we also would pursue treatments for vision loss through the other major cell type of the retina, the photoreceptors,” stated  Brian Culley, Lineage’s CEO. “Our fundamental technology and accumulated know-how give us the opportunity to make many different cell types, and we have demonstrated our ability to create new programs rapidly and efficiently in two distinct areas, expanding our cell therapy pipeline to five separate preclinical and clinical programs, while still maintaining what we believe is an appropriate and responsible rate of investment for a company of our size. This latest program is part of our long-term planning for clinical and commercial success and serves as another example of the capability of our technology platform. We believe our ability to, in just a matter of months, advance from a product concept to generating new intellectual property and manufacturing the desired cell types, is illustrative of the power and efficiency of our platform. We believe the combination of our capital discipline and current balance sheet will support multiple years of further progress, during which we anticipate reaching achievements with each of our clinical and preclinical programs.”

Dr.  Rami Skaliter, who leads the manufacturing function for Lineage, added, “I’m exceptionally proud of the team’s success at overcoming obstacles related to the limited scale of photoreceptor production. Building upon our experience with other cell lineages, we have developed intellectual property, and filed for patent protections, on a manufacturing process which is compatible with large-scale production of photoreceptors in a closed system, improvements which could enable industrial manufacturing. We believe this accomplishment will provide new opportunities for clinical, and ultimately commercial, production of photoreceptors in areas of large unmet need such as Retinitis Pigmentosa, Stargardt’s Macular Dystrophy, and retinal detachments, either independently or through strategic alliances.”

As part of a scientific collaboration with Professors  Benjamin Reubinoff, M.D., Ph.D. and  Eyal Banin, M.D., Ph.D., of the 
Hadassah-Hebrew University Medical Center, the differentiation of pluripotent cells into photoreceptors with clinically compatible characteristics was established utilizing a novel differentiation protocol which generated positive identity of key markers of both rods and cones photoreceptor populations. The data generated by the company further demonstrated that a single cell suspension of photoreceptor precursor cells has the potential to survive and mature post-transplantation in a rodent model of retinal degeneration.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, which is now being developed under a worldwide collaboration with Roche and
Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to (i) the potential amount of payments to Lineage under the alliance with 
Hoffman-La Roche Ltd. (“Roche”) and 
Genentech, Inc., (ii) the potential for new opportunities for clinical, and ultimately commercial, production of photoreceptors in areas of large unmet need, (iii) Lineage’s position to become a leader in the emerging field of regenerative medicine and anti-aging technology, and (iv) future areas of potential treatment using PNC transplant. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the risk that competing alternative therapies may adversely impact the commercial potential of OpRegen, which could materially adversely affect the payments payable to Lineage under the Roche/
Genentech collaboration and license agreement, the risk that Roche/
Genentech may not be successful in completing further clinical trials for OpRegen and/or obtaining regulatory approval for OpRegen in any particular jurisdiction; the risk that Lineage might not succeed in developing products and technologies that are useful in medicine and demonstrate the requisite safety and efficacy to achieve regulatory approval in accordance with its projected timing, or at all; the risk that Lineage’s intellectual property may be insufficient to protect its assets; risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the 
Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the 
SEC, including Lineage’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the 
SEC and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Mike Biega
(Mbiega@soleburytrout.com)
(617) 221-9660

Russo Partners – Media Relations
Nic Johnson or  David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Source: 
Lineage Cell Therapeutics, Inc.